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For instance, if "Biomarkers" and "Genetics" are selected in the Topic category, and "Multiple sclerosis" is selected in the Disease category, all results that match EITHER the Biomarkers OR Genetics Topic category will be shown, but only if they also match the Multiple sclerosis Disease category; i.e. Boolean search of [("Biomarkers" OR "Genetics") AND "multiple sclerosis"]

Scientists CREATE New Gene Therapy Technologies. But Can They Deliver For ALS?

A new gene delivery vehicle may help increase the efficacy of gene therapies being developed for ALS. The adeno-associated virus AAV-PHP.eB, developed by a research team led by Benjamin Deverman and Viviana Gradinaru at the California Institute of Technology, penetrates the central nervous system, including neurons and astrocytes.

A delivery for motor neurons in people with ALS? The vector AAV-PHP.eB delivers genes throughout the brain (top row) and spinal cord (bottom row) about 2 times more efficiently than AAV-PHP.B according to a preclinical analysis. A second gene delivery vehicle, known as AAV-PHP.S, which can be administered simultaneously, penetrates the peripheral nervous system, including the muscles. [Courtesy of Chan et al., 2017, Nature Neuroscience. Reproduced with permission.]

What’s more, the vector, derived from AAV-PHP.B, can transduce more than 50% of these cells using at least 10-fold lower doses (about 4 to 5 x 1012 vector genomes/ kg) according to a preclinical analysis, and is therefore more suitable for clinical applications.

The gene delivery vehicle, identified by a capsid selection method known as Cre recombinase-based AAV selection, is one of a growing number of vectors being developed to deliver potential therapies for neurological diseases in the brain and spinal cord (see June 2017 news). This selection strategy, known as CREATE, is the same approach being used, in collaboration with Boston area startup Voyager Therapeutics in Massachusetts, to develop vectors for a potential treatment for SOD1-linked ALS. The gene therapy, known as VY-SOD101, is at the preclinical stage. The researchers previously used CREATE to identify AAV-PHP.B, a vector that is estimated to be at least 40-fold more efficient than the emerging gene therapy vehicle AAV9 in delivering genes into the brain and spinal cord (Deverman et al., 2016; see June 2017 news).

Meanwhile, in France, researchers are developing a new gene-targeted approach to reduce motor neuron toxicity in SOD1 ALS. The AAV10-based strategy, which involves an injection into the brain and the vasculature, silences the SOD1 gene using a U7-mediated exon skipping mechanism (see May 2017 conference news; Biferi et al., 2017). The approach, according to a 2017 analysis, increases the lifespan of SOD1 G93A mice more than 2-fold than existing RNA interference approaches. Now, Barkats team is adapting this strategy to develop gene therapies for C9orf72 ALS, the most common form of the disease.

Filter articles published since 2015 by topic, disease, or article type.

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